Trial · NCT00744042
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
NCT00744042, Phase 1, Phase 2, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2011-09-30
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP). Trials, record NCT00744042. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00744042
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
- NCT number
- NCT00744042
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 11 participants, actual
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Conditions
- Hypophosphatasia (HPP)
- Interventions
- asfotase alfa
- First posted
- 2008-08-29
- Last updated
- 2019-04-01
- Results first posted
- 2011-09-30
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in Rickets Severity From Baseline to Week 24, Based on Assessment of Skeletal Radiographs Using Radiologic Global Impression of Change (RGI-C) | 24 weeks | A 7-point RGI-C (Radiographic Global Impression of Change) score was used to rate change in rickets severity. Scores ranged from -3 (severe worsening of rickets) to +3 (complete healing of rickets). Only those patients with a minimum score of +2 indicating substantial healing of rickets) were considered "responders". Three pediatric radiologists not affiliated with the conduct of the study performed the ratings. Average scores were derived for each patient at each assessment. |
| Secondary | Maximum Serum Concentration of Asfotase Alfa (Cmax) | Study Week 1 (0 to 168 hours post-dose). Study Week 2 and Study Week 3 (0 to 48 hours post-dose) | Maximum serum concentration observed during intensive PK sampling interval. |
| Secondary | Time at Maximum Serum Concentration of Asfotase Alfa (Tmax) | Study Week 1 (0 to 168 hours post-dose). Study Week 2 and Study Week 3 (0 to 48 hours post-dose). | Time at maximum serum concentration observed during intensive PK sampling interval. |
| Secondary | Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) | Study Week 1 (0 to 168 hours post-dose). Study Week 2 and Study Week 3 (0 to 48 hours post-dose). | Area under serum concentration-time curve to last measurable concentration during intensive PK sampling interval. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in Rickets Severity From Baseline to Week 24, Based on Assessment of Skeletal Radiographs Using Radiologic Global Impression of Change (RGI-C) | 24 weeks | ITT (intention to treat) | Units on a scale | MEDIAN |
| SECONDARY | Maximum Serum Concentration of Asfotase Alfa (Cmax) | Study Week 1 (0 to 168 hours post-dose). Study Week 2 and Study Week 3 (0 to 48 hours post-dose) | ITT population. N=number of patients who received a full dose of asfotase alfa and had sufficient data for non-compartmental analysis. | U/L | MEAN |
| SECONDARY | Time at Maximum Serum Concentration of Asfotase Alfa (Tmax) | Study Week 1 (0 to 168 hours post-dose). Study Week 2 and Study Week 3 (0 to 48 hours post-dose). | ITT population. N=number of patients who received a full dose of asfotase alfa and had sufficient data for non-compartmental analysis. | hour | MEAN |
| SECONDARY | Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) | Study Week 1 (0 to 168 hours post-dose). Study Week 2 and Study Week 3 (0 to 48 hours post-dose). | ITT population. N=number of patients who received a full dose of asfotase alfa and had sufficient data for non-compartmental analysis. | h*U/L | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in Rickets Severity From Baseline to Week 24, Based on Assessment of Skeletal Radiographs Using Radiologic Global Impression of Change (RGI-C) | Asfotase Alfa | 2.00 | Full Range 0.00 to 2.33 | Units on a scale | |
| Maximum Serum Concentration of Asfotase Alfa (Cmax) | Study Week 1 Intravenous Dose (2 mg/kg Single Dose) | 2230 | Standard Deviation 1100 | U/L | |
| Maximum Serum Concentration of Asfotase Alfa (Cmax) | Study Week 2 Subcutaneous Dose (1 mg/kg 3x/Week) | 376 | Standard Deviation 226 | U/L | |
| Maximum Serum Concentration of Asfotase Alfa (Cmax) | Study Week 3 Subcutaneous Dose (1 mg/kg 3x/Week) | 897 | Standard Deviation 491 | U/L | |
| Time at Maximum Serum Concentration of Asfotase Alfa (Tmax) | Study Week 1 Intravenous Dose (2 mg/kg Single Dose) | 4.3 | Standard Deviation 4.3 | hour | |
| Time at Maximum Serum Concentration of Asfotase Alfa (Tmax) | Study Week 2 Subcutaneous Dose (1 mg/kg 3x/Week) | 29.7 | Standard Deviation 13.2 | hour | |
| Time at Maximum Serum Concentration of Asfotase Alfa (Tmax) | Study Week 3 Subcutaneous Dose (1mg/kg 3x/Week) | 12.0 | Standard Deviation 7.7 | hour | |
| Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) | Study Week 1 Intravenous Dose (2mg/kg Single Dose) | 79800 | Standard Deviation 21700 | h*U/L | |
| Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) | Study Week 2 Subcutaneous Dose (1mg/kg 3x/Week) | 14700 | Standard Deviation 9730 | h*U/L | |
| Area Under Serum Concentration-time Curve to Last Measurable Concentration of Asfotase Alfa (AUCt) | Study Week 3 Subcutaneous Dose (1mg/kg 3x/Week) | 33700 | Standard Deviation 19700 | h*U/L |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Asfotase Alfa | 11 | |
| Overall Study | COMPLETED | Asfotase Alfa | 10 | |
| Overall Study | NOT COMPLETED | Asfotase Alfa | 1 |
Source ClinicalTrials.gov
Adverse events114
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Pyrexia | General disorders | Asfotase Alfa | 5 | 11 | 5 |
| other | Irritability | General disorders | Asfotase Alfa | 4 | 11 | 4 |
| other | Injection site erythema | General disorders | Asfotase Alfa | 2 | 11 | 15 |
| other | Catheter site erythema | General disorders | Asfotase Alfa | 1 | 11 | 1 |
| other | Catheter site rash | General disorders | Asfotase Alfa | 1 | 11 | 1 |
| other | Thrombosis in device | General disorders | Asfotase Alfa | 1 | 11 | 1 |
| other | Chills | General disorders | Asfotase Alfa | 1 | 11 | 1 |
| other | Drug withdrawal syndrome | General disorders | Asfotase Alfa | 1 | 11 | 1 |
| other | Hernia | General disorders | Asfotase Alfa | 1 | 11 | 1 |
| other | Hypothermia | General disorders | Asfotase Alfa | 1 | 11 | 1 |
1 to 10 of 114Source ClinicalTrials.gov
See all 114 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Asfotase Alfa | ||
| Total, other adverse events | Asfotase Alfa | 11 | 11 |
| Total, serious adverse events | Asfotase Alfa | 7 | 11 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00744042
GET https://conductscience.org/api/trials/v1/NCT00744042/events?page=2